WO2023178339A3 - Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector - Google Patents
Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector Download PDFInfo
- Publication number
- WO2023178339A3 WO2023178339A3 PCT/US2023/064673 US2023064673W WO2023178339A3 WO 2023178339 A3 WO2023178339 A3 WO 2023178339A3 US 2023064673 W US2023064673 W US 2023064673W WO 2023178339 A3 WO2023178339 A3 WO 2023178339A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- methods
- cardiomyopathy
- compositions
- tnnt2
- treating
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Ceased
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4716—Muscle proteins, e.g. myosin, actin
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
Priority Applications (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CA3254775A CA3254775A1 (en) | 2022-03-18 | 2023-03-18 | METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR |
| JP2024555341A JP2025509828A (en) | 2022-03-18 | 2023-03-18 | Methods and compositions for treating TNNT2-associated cardiomyopathy with viral vectors |
| EP23771729.3A EP4493702A2 (en) | 2022-03-18 | 2023-03-18 | Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector |
| IL315697A IL315697A (en) | 2022-03-18 | 2023-03-18 | Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector |
| KR1020247034272A KR20250007509A (en) | 2022-03-18 | 2023-03-18 | Methods and compositions for treating TNNT2-associated cardiomyopathy using viral vectors |
| US18/847,734 US20250195694A1 (en) | 2022-03-18 | 2023-03-18 | Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector |
| AU2023234604A AU2023234604A1 (en) | 2022-03-18 | 2023-03-18 | Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector |
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263321264P | 2022-03-18 | 2022-03-18 | |
| US63/321,264 | 2022-03-18 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| WO2023178339A2 WO2023178339A2 (en) | 2023-09-21 |
| WO2023178339A3 true WO2023178339A3 (en) | 2023-11-16 |
Family
ID=88024553
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PCT/US2023/064673 Ceased WO2023178339A2 (en) | 2022-03-18 | 2023-03-18 | Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20250195694A1 (en) |
| EP (1) | EP4493702A2 (en) |
| JP (1) | JP2025509828A (en) |
| KR (1) | KR20250007509A (en) |
| AU (1) | AU2023234604A1 (en) |
| CA (1) | CA3254775A1 (en) |
| IL (1) | IL315697A (en) |
| WO (1) | WO2023178339A2 (en) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025214477A1 (en) * | 2024-04-12 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Treatment of genetic cardiomyopathies with aav gene therapy vectors |
| WO2025226841A1 (en) * | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Gene therapy approach for treating disorders associated with tnnt2 |
| WO2025235807A1 (en) * | 2024-05-10 | 2025-11-13 | Solid Biosciences, Inc. | Compositions and methods to improve graft survival |
Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20140256799A1 (en) * | 2013-02-28 | 2014-09-11 | City Of Hope | REPLICATION CAPABLE rAAV VECTORS ENCODING INHIBITORY siRNA AND METHODS OF THEIR USE |
| US20160108430A1 (en) * | 2013-04-17 | 2016-04-21 | Universitätsklinikum Hamburg-Eppendorf | Gene-therapy vectors for treating cardiomyopathy |
| WO2020198337A1 (en) * | 2019-03-25 | 2020-10-01 | Ohio State Innovation Foundation | Engineered mrna sequences and uses thereof |
| WO2021154923A2 (en) * | 2020-01-29 | 2021-08-05 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
| WO2021163357A2 (en) * | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| WO2023108159A1 (en) * | 2021-12-10 | 2023-06-15 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector |
-
2023
- 2023-03-18 WO PCT/US2023/064673 patent/WO2023178339A2/en not_active Ceased
- 2023-03-18 CA CA3254775A patent/CA3254775A1/en active Pending
- 2023-03-18 IL IL315697A patent/IL315697A/en unknown
- 2023-03-18 AU AU2023234604A patent/AU2023234604A1/en active Pending
- 2023-03-18 JP JP2024555341A patent/JP2025509828A/en active Pending
- 2023-03-18 KR KR1020247034272A patent/KR20250007509A/en active Pending
- 2023-03-18 EP EP23771729.3A patent/EP4493702A2/en active Pending
- 2023-03-18 US US18/847,734 patent/US20250195694A1/en active Pending
Patent Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20140256799A1 (en) * | 2013-02-28 | 2014-09-11 | City Of Hope | REPLICATION CAPABLE rAAV VECTORS ENCODING INHIBITORY siRNA AND METHODS OF THEIR USE |
| US20160108430A1 (en) * | 2013-04-17 | 2016-04-21 | Universitätsklinikum Hamburg-Eppendorf | Gene-therapy vectors for treating cardiomyopathy |
| WO2020198337A1 (en) * | 2019-03-25 | 2020-10-01 | Ohio State Innovation Foundation | Engineered mrna sequences and uses thereof |
| WO2021154923A2 (en) * | 2020-01-29 | 2021-08-05 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
| WO2021163357A2 (en) * | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| WO2023108159A1 (en) * | 2021-12-10 | 2023-06-15 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector |
Non-Patent Citations (2)
| Title |
|---|
| TOWNSEND, PJ ET AL ET AL.: "Human Cardiac Troponin T: Identification of Fetal Isoforms and assignment of the TNNT2 Locus to Chromosome 1q", GENOMICS, vol. 21, no. 2, 15 May 1994 (1994-05-15), pages 311 - 316, XP024796242, DOI: 10.1006/geno.1994.1271 * |
| YAMAGUCHI YUYA, TANIHATA JUN, BABA SHUNSUKE, MORIMOTO SACHIO, MINAMISAWA SUSUMU: "Wild‐type troponin T gene overexpression in mice with troponin T mutant‐induced dilated cardiomyopathy partially rescued cardiac pathology", THE FASEB JOURNAL, FEDERATION OF AMERICAN SOCIETIES FOR EXPERIMENTAL BIOLOGY, US, vol. 35, no. S1, 1 May 2021 (2021-05-01), US, pages 2, XP009550733, ISSN: 0892-6638, DOI: 10.1096/fasebj.2021.35.S1.02274 * |
Also Published As
| Publication number | Publication date |
|---|---|
| AU2023234604A1 (en) | 2024-10-10 |
| CA3254775A1 (en) | 2023-09-21 |
| KR20250007509A (en) | 2025-01-14 |
| WO2023178339A2 (en) | 2023-09-21 |
| EP4493702A2 (en) | 2025-01-22 |
| US20250195694A1 (en) | 2025-06-19 |
| IL315697A (en) | 2024-11-01 |
| JP2025509828A (en) | 2025-04-11 |
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